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Raxone(idebenone)film-coated tablet

ID号：787 发布日期： 2016-07-21 截止日期：不限地区：全国浏览次数：3

欧盟委员会批准Raxone用于Leber遗传性视神经病变
欧盟委员会批准Santhera的首款药物Raxone，用于Leber遗传性视神经病变（LHON）青少年及成年患者的视觉障碍治疗。这种罕见病症是一种遗传性的线粒体疾病，如果不进行治疗，它通常会使健康患者快速、深刻及永久性失明。
我们的数据证明，Raxone治疗可以阻止患者进一步的视力丧失，能够促进临床相关的视力恢复，Santhera首席执行官Meier博士称。对Santhera来说，此次 Raxone作为一款有效的治疗药物在欧洲获批用于LHON及作为首款获批用于线粒体疾病是一个重要的里程碑，因为它标志着我们转变成一个有产品上市的制药公司。
Raxone是线粒体疾病治疗领域的一项重大突破
Raxone 在线粒体疾病治疗领域代表了一项重大突破，它的批准为 LHON 患者获得治疗、实现有意义的视力改善铺平了道路，德国慕尼黑大学神经学教授 Klopstock称，他也是线粒体疾病德国网络的协调者及一名LHON研究者。LHON 是一种特别灾难性的疾病，因为遭受这种疾病的患者不健康，并通常比较年轻，他们在几个月内会迅速双眼失明。如果不进行治疗，多数患者仍会持续失明。
Raxone是一种口服药物，每天的用药剂量为900mg（每天三次，每次2片，随食物一起服用），用于青少年及成年LHON患者视觉障碍治疗。疗效数据基于 Santhera的随机、安慰剂对照RHODOS试验及开放式扩大获取项目，这两项数据一起证明在 Raxone治疗患者中视力丧失是可以缓解或逆转的。
Raxone®(idebenone)150mg film-coated tablet
The European Commission has granted marketing authorization for Raxone (idebenone) for the treatment of visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON).
This rare condition is an inherited mitochondrial disease that can lead to rapid, profound and permanent blindness in otherwise healthy patients. Typically it occurs in young adults but it can occur at any age. The rapid vision loss is believed to be the result of mitochondrial mutations that results a loss of function among several NADH-ubiquinone oxidoreductase chains.
Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1).
Efficacy of Idebenone in Leber's Hereditary Optic Neuropathy
In 2011, Klopstock et al1 reported that the primary endpoint (best recovery in visual acuity) was not achieved in their 24-week multicenter, placebo-controlled, randomized, double-blind study involving 85 patients with LHON. However, numerous secondary measures did reach statistical significance (changes in visual acuity in best eye; changes in visual acuity for both eyes in each patient).
A follow-up study in 60 of the 85 patients, showed a similar pattern of visual improvement that was not statistically significant.2
In a statement3, lead investigator of those studies— Thomas Klopstock, MD, Professor for Neurology at the University of Munich—said, "Raxone represents a major breakthrough in mitochondrial disease treatment, and its approval paves the way for patients with LHON to be treated and to achieve a meaningful improvement of their visual acuity."
Roxane (idebenone) treatment (900 mg daily given as 2 tablets three times a day with food) should be initiated and supervised by a physician with experience in LHON
Approximately 8000 people in Europe have LHON.
Also Being Studied for Duchenne Muscular Dystrophy and Multiple Sclerosis
In the United States, idebenone has orphan designation for LHON but it is not known when an NDA will be filed. The drug is also being investigated for treatment of Duchenne muscular dystrophy (phase 3 study) and multiple sclerosis (phase 2 study).